Overview

Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis

Status:
Completed

Trial end date:
2009-10-01

Target enrollment:

Participant gender:

Summary

Cystinosis is an inheritable disease that if untreated, results in kidney failure as early as the first decade of life. The current marketed therapy is Cystagon® (cysteamine bitartrate) which must be taken every six hours for the rest of the patient's life to prevent complications of cystinosis. RP103 is a formulation of cysteamine bitartrate that is being studied to see if it may be able to be given less frequently, once every 12 hours, and have similar results.

Phase:

Phase 1/Phase 2

Details

Lead Sponsor:

Horizon Pharma USA, Inc.
Raptor Pharmaceuticals Inc.

Treatments:

Cysteamine